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Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio.
Waldrop, Megan A; Chagat, Shannon; Storey, Michael; Meyer, Alayne; Iammarino, Megan; Reash, Natalie; Alfano, Lindsay; Lowes, Linda; Noritz, Garey; Prochoroff, Andre; Rossman, Ian; Ginsberg, Matthew; Mosher, Kathryn; Broomall, Eileen; Bass, Nancy; Gushue, Courtney; Kotha, Kavitha; Paul, Grace; Shell, Richard; Tsao, Chang-Yong; Mendell, Jerry R; Connolly, Anne M.
Afiliación
  • Waldrop MA; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA; Departments of Pediatrics and Neurology, Ohio State University Wexner Medical Center, Columbus OH, 43205, USA. Electronic address: megan.waldrop@nationwidechildrens.org.
  • Chagat S; Department of Neurology, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Storey M; Department of Pharmacy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
  • Meyer A; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA.
  • Iammarino M; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA.
  • Reash N; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA.
  • Alfano L; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA.
  • Lowes L; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA.
  • Noritz G; Department of Pediatrics, Nationwide Children's hospital, Columbus, OH 43205, USA.
  • Prochoroff A; Department of Pediatric Neurology, Metro Health, Cleveland OH, USA.
  • Rossman I; Department of Pediatric Neurology, Akron Children's Hospital, Akron OH, USA.
  • Ginsberg M; Department of Pediatric Neurology, Akron Children's Hospital, Akron OH, USA.
  • Mosher K; Department of Pediatric Physiatry, Akron Children's Hospital, Akron OH, USA.
  • Broomall E; Department of Pediatric Neurology, Cincinnati Children's Hospital Medical Center, Cincinnati OH, USA.
  • Bass N; Division of Pediatric Neurology, Department of Pediatrics, Rainbow Babies and Children's Hospital, Cleveland OH, USA.
  • Gushue C; Departments of Pediatrics and Pulmonology, Ohio State University Wexner Medical Center, Columbus OH 43205, USA.
  • Kotha K; Departments of Pediatrics and Pulmonology, Ohio State University Wexner Medical Center, Columbus OH 43205, USA.
  • Paul G; Departments of Pediatrics and Pulmonology, Ohio State University Wexner Medical Center, Columbus OH 43205, USA.
  • Shell R; Departments of Pediatrics and Pulmonology, Ohio State University Wexner Medical Center, Columbus OH 43205, USA.
  • Tsao CY; Departments of Pediatrics and Neurology, Ohio State University Wexner Medical Center, Columbus OH, 43205, USA.
  • Mendell JR; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA; Departments of Pediatrics and Neurology, Ohio State University Wexner Medical Center, Columbus OH, 43205, USA.
  • Connolly AM; Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus OH, 43205, USA; Departments of Pediatrics and Neurology, Ohio State University Wexner Medical Center, Columbus OH, 43205, USA.
Neuromuscul Disord ; 34: 41-48, 2024 Jan.
Article en En | MEDLINE | ID: mdl-38142474
ABSTRACT
5q spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease caused by absence of the SMN1 gene with three FDA approved genetic therapies which significantly improve outcomes. The AAV9 mediated gene replacement therapy, onasemnogene abeparvovec, has the greatest potential for side effects. Here we report the safety and outcomes from 46 children treated with onasemnogene abeparvovec in the state of Ohio between December 2018 and January 2023. In our cohort, onasemnogene abeparvovec treatment remained safe and no child experienced any significant adverse events, including thrombotic microangiopathy, liver failure or death. All children experienced benefit, although the benefit in those with 2 copies of SMN2 was variable. 79 % of the children treated when symptomatic had a SMN2 modifying therapy added on. With careful screening and post treatment monitoring, onasemnogene abeparvovec is safe and effective for children with SMA in the state of Ohio, but more work needs to be done to ensure optimal outcomes for all children with 2 copies of SMN2.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Productos Biológicos / Proteínas Recombinantes de Fusión / Atrofia Muscular Espinal / Atrofias Musculares Espinales de la Infancia / Enfermedades Neurodegenerativas Límite: Child / Humans País/Región como asunto: America do norte Idioma: En Revista: Neuromuscul Disord Asunto de la revista: NEUROLOGIA Año: 2024 Tipo del documento: Article Pais de publicación: Reino Unido
Texto completo
Añadir a Mi BVS
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PubMed Links
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Productos Biológicos / Proteínas Recombinantes de Fusión / Atrofia Muscular Espinal / Atrofias Musculares Espinales de la Infancia / Enfermedades Neurodegenerativas Límite: Child / Humans País/Región como asunto: America do norte Idioma: En Revista: Neuromuscul Disord Asunto de la revista: NEUROLOGIA Año: 2024 Tipo del documento: Article Pais de publicación: Reino Unido